Unmet needs, underfunded science: A call for investment in cerebral palsy research across the lifespan.

Cerebral palsy (CP) research has broadened its horizons in the past decades. In addition to the long-term, population-based studies of prevalence of the different CP types, gestational-age groups, and accompanying impairments, data on several aspects of function have emerged. The well-accepted Gross Motor Function Classification System1 has given us a common language to compare gross motor function in different populations and at various ages. Gross motor classification has been followed by classifications of hand function, such as the Bimanual Fine Motor Function2 and the Manual Ability Classification System.3 However, differences in CP classification around the world still exist. This is well illustrated by Rice et al. in their report from the South Australian CP Register in the current issue of the journal. Although a method of classification was proposed along with the most recent definition of CP,4 the traditional systems tend to predominate. This makes comparison between CP registers difficult and important knowledge about trends, differences, and similarities may be overlooked or lost. An essential aspect of the diagnosis of CP is the neurological signs. It is now recommended that classification should be made according to the dominant type of tone or movement abnormality. This has not been the case before in all countries, and has made any attempt to pool data difficult. However, such comparisons have been made possible in Europe after the introduction of the Reference and Training Manual, produced by the Surveillance of Cerebral Palsy in Europe (SCPE). It contains descriptions and video illustrations of the different CP types. It is an excellent tool in the endeavor to harmonize classification of CP in Europe and elsewhere.5 The paper by Rice et al. is a commendable attempt to carry out a comparison between CP registers on two different continents, despite differences in classification systems. The fact that this is feasible is very promising, but the paper also depicts the difficulties of such a comparison. This is especially apparent when comparing the quadriplegia and dyskinetic groups in the Australian and Swedish registers. Although very similar groups are described the outcome regarding classification differed, because of the different traditions in the two countries on classifying dyskinetic symptoms. The prevalence of dyskinetic CP may be different from what we find in most studies today if, or when, classification according to the dominating symptom is fully accepted. As we strive to describe the symptoms and function of children with CP, we have to accept that there are not yet valid and reliable instruments for every measurement. When there is such an instrument is at hand, it should be used to compare studies, and also to make it possible for other researchers to relate to the data. The specific differentiation between dyskinetic and spastic features may indeed be a challenge, as will be, in some cases, deciding which is in fact the dominating symptom. Children with dyskinetic CP, classified according to dominating movement abnormality, have in a majority of cases also signs of spasticity.6 Very few instruments exist to describe dyskinesia. As dyskinetic movements may severely hamper motor function, there is a need for valid and reliable methods to assess this aspect of motor disability. The Barry-Albright Dystonia Scale, referred to by Rice et al., deals with the dystonic, but not the hyperkinetic symptoms.7 This scale is seldom used in CP research, but might be considered a useful tool.6 Also, in children with predominantly spastic CP there may be involuntary movements of a choreo-athetotic type that interfere with activities, and we have little means to describe this phenomenon in a structured way. Despite all this, Rice et al. take on the challenge to describe and discuss the dyskinetic features in children with CP and find this in almost every fifth child! CP research is carried out in various ways around the world. Many CP registers rely on reports from local physicians on what CP type, gross and fine motor function, and accompanying impairments a child might have, while others, such as the South Australian CP Register, has the advantage of a clinical examination in addition to the data from the records. Here is an opportunity to apply a variety of assessment instruments to ascertain information that is not possible to assess from records, but that is required to investigate the child. This may be one way to develop further population-based CP research. Future studies from the South Australian CP register will surely benefit from the access to clinical investigation using valid and reliable instruments to gather more population-based knowledge on CP.

Purpose Identify perceptions of parents and caregivers of children with cerebral palsy about being consumer research partners and identify strategies to inform involvement of parents in cerebral palsy research. Materials and methods Twenty-two parents in New South Wales and Victoria (Australia) participated in this qualitative study. Seven interviews and three focus groups were completed. Interpretive description guided data analysis. Methodological rigor was enhanced through involving two consumer investigators in the research team, member checking, and multiple researchers completing data analysis and theme generation. Results Participants identified a range of factors that may influence their involvement in research partner roles. Main topics emerging from the data included “Research Is Better with Parents” and “Parents Benefit from Being Research Partners.” A third, “Parents as Research Partners,” contained the themes “Flexible Involvement,” “Starting Partnerships,” and “Building and Sustaining Partnerships.” Conclusion This study has provided a rich insight into how parents perceive and describe engaging as research partners. Parent-identified guidance will inform future partnerships aiming to enhance the quality of cerebral palsy research and outcomes for people with cerebral palsy and their families. The involvement of consumer investigators in this study was considered valuable for enhancing the quality and applicability of the research. IMPLICATIONS FOR REHABILITATION Parents believed that parent partnership in research has benefits for the research and for the consumers involved. Parents provided guidance about the importance of starting, building and sustaining relationships in involving parents as research partners. Understanding the parent context, investing in relationships and acknowledgement of, and recognition for, contributions were considered important for building and sustaining effective partnerships. Flexible approaches to supporting parents as research partners was considered necessary for effective partnership.

Cerebral palsy (CP) describes a heterogeneous group of non-progressive neurodevelopmental disorders affecting movement and posture. The etiology and diagnostic biomarkers of CP are a hot topic in clinical research. Recent advances in omics techniques, including genomics, epigenomics, transcriptomics, metabolomics and proteomics, have offered new insights to further understand the pathophysiology of CP and have allowed for identification of diagnostic biomarkers of CP. In present study, we reviewed the latest multi-omics investigations of CP and provided an in-depth summary of current research progress in CP. This review will offer the basis and recommendations for future fundamental research on the pathogenesis of CP, identification of diagnostic biomarkers, and prevention strategies for CP.

Background Cerebral palsy has been linked to decreased quality of life. However use of self- versus proxy-perspectives and norm-sample comparisons to examine quality of life impact may produce different results. Aims To compare quality of life ratings in children and adolescents with cerebral palsy relative to typically developing peers in consideration of sample and methodological moderators on estimated effects. Methods Eleven independent studies, comprising a pooled sample of 1475 families living with cerebral palsy and 42119 peers, were identified. Study reporting quality was evaluated with the QualSyst tool and standardised mean group differences (Hedges’ g) with associated confidence intervals and p values calculated. Heterogeneity was examined using a random effects model. Results All studies provided good to excellent methodological and statistical detail. Physical quality of life was significantly impaired among those with cerebral palsy (g range: −0.42 to −1.58). However, inconsistent findings were noted in relation to the effect of cerebral palsy on psychological (g range: 0.04 to −0.80) and social quality of life (g range: −0.80 to −0.51), depending on the measurement used. There was a trend for parents to evaluate their child’s physical quality of life lower than child-reported scores. Conclusions Physical quality of life is, invariably, more affected in those with cerebral palsy. The connection between cerebral palsy and psychosocial quality of life is less clear. Noted parent-child discrepancies highlight the value of a multi-informant approach to child quality of life assessment. Implications for rehabilitation Quality of life is an important health-related outcome in cerebral palsy research and practice. Collecting both self-report and proxy data can help to highlight quality of life issues that are salient to the parent and to the child or adolescent with cerebral palsy. Selection of the appropriate quality of life instrument depends on the assessment purpose, with available measures varying in their focus on functionality, subjectivity and illness-specific items. Quality of life assessment of children with cerebral palsy should extend beyond functional abilities to include less obvious, but critical, psychological and social issues.

Stakeholder Perspectives on Engaging With Cerebral Palsy Research Studies After Onset of COVID-19 in the United States

Objective(s)Our objective was to investigate the motivators and barriers associated with the individual or family decision to participate in cerebral palsy research. Based on this information, we offer suggestions to increase the likelihood of participation in future CP studies.MethodsA digital survey was administered to stakeholders affected by cerebral palsy across the US. Our analysis focused on variables related to personal interests, travel, and study-specific elements. Statistical tests investigated the effects of responder type, cerebral palsy type, and Gross Motor Function Classification System level on travel and study-specific element variables. Recommendations were informed by responses reflecting the majority of respondents.ResultsBased on 233 responses, we found that respondents highly valued research participation (on average 88.2/100) and compensation (on average 62.3/100). Motivators included the potential for direct benefit (62.2%) and helping others (53.4%). The primary barriers to participation were schedule limitations (48.9%) and travel logistics (32.6%). Schedule limitations were especially pertinent to caregivers, while individuals with more severe cerebral palsy diagnoses reported the necessity of additional items to comfortably travel.ConclusionsOverall, we encourage the involvement of stakeholders affected by cerebral palsy in the research process. Researchers should consider offering flexible study times, accommodating locations, and compensation for time and travel expenses. We recommend a minimum compensation of $15/hour and a maximum time commitment of 4 hours/day to respect participants’ time and increase likelihood of research participation. Future studies should track how attitudes toward research change with time and experience.

Our objective was to investigate the motivators and barriers associated with the individual or family decision to participate in cerebral palsy research. Based on this information, we offer suggestions to increase the likelihood of participation in future CP studies. A digital survey was administered to stakeholders affected by cerebral palsy across the US. Our analysis focused on variables related to personal interests, travel, and study-specific elements. Statistical tests investigated the effects of responder type, cerebral palsy type, and Gross Motor Function Classification System level on travel and study-specific element variables. Recommendations were informed by responses reflecting the majority of respondents. Based on 233 responses, we found that respondents highly valued research participation (on average 88.2/100) and compensation (on average 62.3/100). Motivators included the potential for direct benefit (62.2%) and helping others (53.4%). The primary barriers to participation were schedule limitations (48.9%) and travel logistics (32.6%). Schedule limitations were especially pertinent to caregivers, while individuals with more severe cerebral palsy diagnoses reported the necessity of additional items to comfortably travel. Overall, we encourage the involvement of stakeholders affected by cerebral palsy in the research process. Researchers should consider offering flexible study times, accommodating locations, and compensation for time and travel expenses. We recommend a minimum compensation of $15/hour and a maximum time commitment of 4 hours/day to respect participants' time and increase likelihood of research participation. Future studies should track how attitudes toward research change with time and experience.

Dean, colleagues, I am very honoured by your invitation to give the Duncan lecture for 1997. In speaking of Dr Duncan’s legacy in a remote New Guinea valley, I would...

This commentary is on the original article by Salami et al. on pages 833–838 of this issue.

This commentary is on the original article by Cheong et al. on pages 655–660 of this issue.

ABSTRACTBackgroundCerebral palsy (CP) clinical research is fraught with challenges, in part due to health‐related disparities common among people with disabilities. Perspectives of people with lived experience of CP, clinicians and researchers vary on how to address these disparities. The present initiative explores synergies and discrepancies among stakeholders (n = 212) representing these partner groups in perceived barriers and facilitators to high‐quality clinical CP research and robust trainee pathways. The overarching goal is to generate priority actions to empower meaningful partner group engagement in CP research and, ultimately, improve health outcomes for people with CP.MethodsGrounded in empowerment theory, mixed methods needs assessments were conducted separately with partner groups to capture perspectives on barriers and facilitators to high‐quality CP research and strong trainee pathways. Thematic analysis was applied to focus groups and interviews to identify themes and subthemes.ResultsDiscrepancies among partner groups emerged related to informational needs, community connection, ethical research and equitable representation in research, and fair compensation for lived experience partner engagement in the research process.ConclusionsOngoing opportunities for researcher action to empower partner group engagement include building shared purpose, nurturing social connection within and among groups and intentional efforts to build trust and codesign studies.Patient or Public ContributionThe initiative described here was informed by caregivers of children with CP from Georgia, USA, using a community‐based participatory research (CBPR) approach. CPBR is a collaborative approach, designed to give communities, which here include people with lived experience of CP, control over research processes and outcomes. Their perspectives were essential to the premise of this study and guided data interpretation, especially with regard to how their perspectives may or may not correspond to those of CP researchers and clinicians. To ensure inclusion of all perspectives, individuals with CP were also represented in these latter two engagement groups. Finally, the design, conduct, analysis and interpretation of data were informed by a researcher and a clinician‐scientist, both of whom have lived experience as caregivers of children with CP.

The criterion standard for research in all medical fields is the randomized clinical trial (RCT). Done correctly, this design enables the researcher to pose a question prospectively, assemble two groups – one of which is a control group and the other the experimental group – and determine if the intervention leads to differences between the two. Since RCTs are so highly valued, why are there so few of them in our field?1 Firstly, there are several problems in creating large, informative, comparable groups. Secondly, the cost for large double-blind RCTs is huge, which restricts research to major centers that have the necessary expertise and infrastructure; it also restricts access to major grants from governments or industry (the latter possibly inducing biases).2 Third, generalizability of the results to individuals who have one of the exclusion criteria may be difficult. So what should we do in the field of cerebral palsy (CP) and other developmental disabilities to evaluate effectiveness?2 The answer might be clinical effectiveness research (CER) – perhaps the future of our field? Simply stated, CER is a method in which one treats a group of patients the way that one thinks is best for the particular patients’ issues. The difference between this and regular clinical practice is that there a systematic way, using validated instruments, to collect data prior to intervention and at routine intervals after the intervention. Various statistical techniques are applied and research questions can be answered. This is ‘real world’ research, without any inclusion or exclusion criteria. In order to house all these data, one must have a repository of the information, hence the huge interest in registers. A register (or registry) involves enrolling a population in a computerized database with a prescribed set of common data elements. At regular intervals, usually when there is some sort of intervention, another set of validated instruments is used to evaluate the patients. Participation of many centers would result in a very large database to study. With a register, one could query the database to find, for example, all of the patients with bilateral CP who had mild dystonia, upper extremity contractures and seizure disorders. By comparing different approaches (oral medications, botulinum toxin, surgery, therapies) it is possible to tease out the factors that made one approach more successful than the others. The use of CER and registers is not as methodologically rigorous as the RCT, but given the challenges of the RCT, CER and registers provide counterpoints. In theory, all the patients in one's practice could be enrolled. The results of these studies are generalizable (to the patient you are treating); the studies are relatively inexpensive (still very expensive, but not on the scale of a large RCT); and the research does not have to be performed in large universities, but can be done in smaller community settings. This type of study also allows one to follow a patient for years – essential in our patient population – rather than until grant money runs out! It does take a lot of investigator time to enroll and continue to follow a subject, but for those who have been involved in registers, the research output is tremendous. Certainly, I do not advocate eliminating randomized double-blind clinical trials. However, to move our field forward, we must utilize CER and registers to obtain a more generalizable real-world understanding of the natural history and what ‘works’ in our field.

Cerebral palsy (CP), a common pediatric movement disorder, causes the most severe physical disability in children. Early diagnosis in high-risk infants is critical for early intervention and possible early recovery. In recent years, multivariate analytic and machine learning (ML) approaches have been increasingly used in CP research. This paper aims to identify such multivariate studies and provide an overview of this relatively young field. Studies reviewed in this paper have demonstrated that multivariate analytic methods are useful in identification of risk factors, detection of CP, movement assessment for CP prediction, and outcome assessment, and ML approaches have made it possible to automatically identify movement impairments in high-risk infants. In addition, outcome predictors for surgical treatments have been identified by multivariate outcome studies. To make the multivariate and ML approaches useful in clinical settings, further research with large samples is needed to verify and improve these multivariate methods in risk factor identification, CP detection, movement assessment, and outcome evaluation or prediction. As multivariate analysis, ML and data processing technologies advance in the era of Big Data of this century, it is expected that multivariate analysis and ML will play a bigger role in improving the diagnosis and treatment of CP to reduce mortality and morbidity rates, and enhance patient care for children with CP.

The purpose of this study was to measure the growth of the Cerebral Palsy (CP) Research Network towards becoming a Learning Health Network in order to guide future development. Thirteen CP Research Network leaders completed the Network Maturity Grid (NMG) which consists of six domains with eight to 10 components each. The six domains are Systems of Leadership, Governance and Management, Quality Improvement, Engagement and Community, Data and Analytics, and Research. Radar mapping was utilized to display mean scores on a 5-point ordinal scale (1 = not started to 5 = idealized state) across domains and for individual components within domains. Consensus was reached for top priorities for the next 3-5 years. Domain scores ranged from 2.4 in Quality Improvement to 3.2 in System of Leadership. The lowest scoring component was clinician clinical decision support and the highest was common purpose. The following priority areas of focus were agreed upon moving forward: development of leaders, financial sustainability, quality improvement education and training, patient reported data, data quality and validation, and primary data collection. Results from this project will be utilized for strategic planning to improve the network. Conducting regular self-assessments of the network with the NMG will be useful in achieving the network's ultimate goal to improve care and outcomes for individuals with CP.

Advancements in the treatment of cerebral palsy depend on animal research. Yet, most animal models have not been fully evaluated for spasticity and dystonia using clinically relevant measures of altered tone or movement patterns, which form the basis for diagnosing people with the condition. Sensory differences and pain are almost never studied in animal models. Complicating factors include the diversity of animals and injuries used to model the condition, the diversity of outcomes after acquired injury, and translating clinical measures into reliable and repeatable measurements tailored to animals, ideally using common data elements. We summarize preclinical models based on acquired injury to the nervous system in cerebral palsy research over the years and provide a comparison of developmental time courses for common laboratory animals. We encourage researchers to coalesce on consistent, reliable measurements for assessing both sensory and motor systems to ensure that animal models reflect meaningful aspects of the condition.